Forget Designer Babies. Here’s How CRISPR is Really Changing Lives
upstart writes:
The gene-editing tool is being tested in people, and the first treatment could be approved this year:
Forget about He Jiankui, the Chinese scientist who created gene-edited babies. Instead, when you think about gene editing you should think of Victoria Gray, the African-American woman who says she's been cured of her sickle-cell disease symptoms.
[...] But the designer-baby debate is a distraction from the real story of how gene editing is changing people's lives, through treatments used on adults with serious diseases.
In fact, there are now more than 50 experimental studies underway that use gene editing in human volunteers to treat everything from cancer to HIV and blood diseases, according to a tally shared with MIT Technology Review by David Liu, a gene-editing specialist at Harvard University.
Most of these studies-about 40 of them-involve CRISPR, the most versatile of the gene-editing methods, which was developed only 10 years ago.
[...] To scientists, CRISPR is a revelation because of how it can snip the genome at specific locations. It's made up of a cutting protein paired with a short gene sequence that acts like GPS, zipping to a predetermined spot in a person's chromosomes.
[...] The first generation of CRISPR treatments are also limited in another way. Most use the tool to damage DNA, essentially shutting off genes-a process famously described as "genome vandalism" by Harvard biologist George Church.
[...] Liu's lab is working on next-generation gene-editing approaches. These tools also employ the CRISPR protein, but it's engineered not to cut the DNA helix, but instead to deftly swap individual genetic letters or make larger edits. These are known as "base editors."
[...] Now that gene editing has had its first successes, Urnov says, there's an "urgent need" to open a "path to the clinic for all."
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